PTC Therapeutics, Inc. (NASDAQ: PTCT) has announced that Waylivra has obtained Category 1 classification from the Câmara de Regulaço do Mercado de Medicamentos – CMED (Drug Market Regulation Chamber) in Brazil.
Waylivra is the sole FCS therapeutic alternative in Brazil
Waylivra is the only therapeutic option for familial chylomicronemia syndrome (FCS) in Brazil. Innovative medicines that provide more efficacy than existing standards of care are classified as Category 1, which permits for pricing inconsistent with global markets.
Chief Executive Officer of PTC Therapeutics Stuart W. Peltz said, “Both FCS and FPL are rare genetic diseases that cause significant issues for patients suffering from these disorders. I am pleased with the decision by CMED to grant FCS patients access to Waylivra in Brazil. This innovative treatment will have a positive impact on patients’ lives. I am also very enthusiastic about the possibility of bringing Waylivra to patients suffering from FPL who are also in need of treatment.”
PTC also applied to the Health Regulatory Agency of Brazil, Agência Nacional de Vigilância Sanitária (ANVISA), to have Waylivra approved for familial partial lipodystrophy (FPL) treatement. If authorized, Waylivra is the first medication for FPL to be approved in Brazil, and it will also be the first approval for this indication globally. The application was submitted under the RDC205/2017 Rare Disease route. According to PTC, the regulatory authorities are expected to make a decision in the second half of 2022.
FCS can cause pancreatitis and other chronic problems
FCS is a rare genetic illness that causes severe disease load in patients, including potentially deadly pancreatitis and chronic consequences from permanent organ damage. FPL is a rare hereditary illness that causes the sufferer to experience both visual and bodily anguish. It causes serious metabolic problems that aren’t addressed by current treatments.
Waylivra is an Ionis Pharmaceuticals novel antisense tech granted conditional marketing approval in Europe for FCS treatment. Additionally, Waylivra has orphan drug designation from the EMA for the indication and is licensed from Akcea Therapeutics, a subsidiary of Ionis.